With an eye toward 2020, CIRM looks at clinical milestones achieved in 2016

One year ago, CIRM announced its strategic plan for the next five years. It’s a bold vision to maximize our impact in stem cell research by accelerating stem cell treatments to patients with unmet medical needs.

Our strategic plan, which can be found on our website, details how CIRM will invest in five main program areas including infrastructure, education, discovery, translation and clinical research. While CIRM has invested in these areas in the past, we are doing so now with a renewed focus to make sure our efforts have a lasting impact in California and more importantly for patients.

Now that a year has passed, it’s time to review our progress and look ahead to the next four years.

Our Progress

2016 was a very productive year. On the infrastructure side, CIRM successfully launched the Translating and Accelerating Centers, awarding both grants to QuintilesIMS. The Translating Center supports preclinical research that’s ready to advance to clinical trials but still needs approval by the US Food and Drug Administration (FDA). The Accelerating Center picks up where the Translating Center leaves off and offers support and management services for clinical trial projects to ensure that they succeed. Collectively called The Stem Cell Center, the goal of this new infrastructure is to increase efficiency and shorten the time it takes to get human stem cell trials up and running.

On the research side in 2016, CIRM funded over 70 promising stem cell projects ranging from education to discovery, translational and clinical projects. While of these areas are important to invest in, CIRM has shifted its focus to funding clinical trials in hopes that one or more of these trials will develop into an approved therapy for patients. So far, we’ve funded 25 trials, 22 of which are currently active since CIRM was established.

In our strategic plan, we gave ourselves the aggressive goal of funding 50 new clinical trials by 2020, which equates to 10 new trials per year. So far in 2016, we’ve funded eight clinical trials and tomorrow at our December ICOC meeting, our Governing Board will determine whether we meet our yearly clinical milestone of 10 trials by considering two more for funding.

The first trial is testing a stem cell treatment that could improve the outcome of kidney transplants. For normal kidney transplants, the recipient is required to take immunosuppressive drugs to prevent their body from rejecting the donated organ. This clinical trial aims to bypass the need for these drugs, which carry an increased risk of cancer, infection and heart disease, by injecting blood stem cells and other immune cells from the kidney donor into the patient receiving the kidney. You can read more about this proposed trial here.

The second clinical trial is a stem cell derived therapy to improve vision in patients with a degenerative eye disease called retinitis pigmentosa. This disease destroys the light sensing cells at the back of the eye and has no cure. The trial hopes that by transplanting stem cell derived retinal progenitor cells into the back of the eye, these injected cells will secrete factors that will keep the cells in the eye healthy and possibly improve a patient’s vision. You can read more about this proposed trial here.

Our Future

No matter the outcome at tomorrow’s Board meeting, I think our agency should be proud of its accomplishments since launching our strategic plan. The eight clinical trials we’ve funded this year are testing stem cell therapies for diseases including muscular dystrophy, kidney disease, primary immune diseases, and multiple types of cancer and blood disorders.

At this pace, it seems likely that we will achieve many of the goals in our strategic plan including our big goal of 50 new clinical trials. But pride and a sense of accomplishment are not what CIRM is ultimately striving for. Our mission and the reason why we exist are to help people and improve their lives. I’ll leave you with a quote from our President and CEO Randy Mills:

Randy Mills

“In everything we do there is a real sense of urgency, because lives are at stake. Our Board’s support for these programs highlights how every member of the CIRM team shares that commitment to moving the most promising research out of the lab and into patients as quickly as we can.”

Stem cell therapy seems like the answer to the disease process, yet it is so slow to finally get to the public for realistic use. There are so many complicated regulations in all the types of trials, i.e., single arm, double, placebo, etc. etc. I am a little disheartened I will never see a use personally as it is so slow.

We understand your feelings about the slow pace of getting approved stem cell therapies to patients who need them. That is why it is important for funding agencies like CIRM to continue to fund more promising stem cell therapies in clinical trials. The more trials we fund, the more likely a therapy will succeed and be approved for use. There will likely be changes in the regulations of stem cell therapies in the future with the passing of the Cures Act by Congress. Hopefully this will allow for the faster approval of safe and effective stem cell therapies for patients and not therapies whose safety is still under question.

Thank you for bringing up the Cures act, and worth noting that the first thing mentioned by the VP is cancer immunotherapy. I’m very curious how everything will shake out, and hopeful that an “inject and see” attitude does not dominate, but a focus on increased resources from the FDA enable milestone achievement.

Hi Shaun, I did not mean to criticize, just that it seems like practical application is so slow….in a perfect world there would be unlimited funding and no FDA! I am an RN and deal with medical issues as well…..I have RP and have been to two of Dr. Klassen’s presentations hosted by FFB, and am aware of all the research CIRM has contributed to this huge project. With all the protocols that must be met, it really drags things out, and a lot of patients with cancer certainly have priority over a chronic disease process. Keep up the good work, it IS APPRECIATED.